2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 b-thalassemia patient cells restoring hemoglobin A production and chain rebalance

Alisa Dong, Valentina Ghiaccio, Irene Motta, Shuling Guo, Raechel Peralta, Susan M. Freier, Andy Watt, Sagar Damle, Yasuhiro Ikawa, Danuta Jarocha, Maxwell Chappell, Coralea Stephanou, Paola Delbini, Connie Chen, Soteroula Christou, Marina Kleanthous, Kim Smith-Whitley, Deepa Manwani, Carla Casu, Osheiza AbdulmalikMaria Domenica Cappellini, Stefano Rivella, Laura Breda

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Medicine & Life Sciences